Form 8-K uniQure N.V. For: Jun 21

June 23, 2021 6:05 AM EDT

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Exhibit 99.1

 

 

uniQure to Acquire Corlieve Therapeutics and Advance its
Gene Therapy Program to Treat Temporal Lobe Epilepsy (TLE)

 

~ Expands uniQure’s Pipeline of Innovative Gene Therapies to Treat Neurological Disorders ~

 

~ Strengthens uniQure’s Global Leadership in the Development of Gene Therapies that Employ miRNA 

Silencing Technology ~

 

~ Large Opportunity Targeting an Estimated 1.3 million TLE Patients in the U.S. and Europe with
Approximately 800,000 Drug-Resistant Patients ~

 

~ Preclinical Proof-of-Concept Data from Disease Model and from Resected Hippocampi from Refractory TLE
Patients Demonstrate Clear Suppression of Chronic Spontaneous Epileptic Seizures ~

 

Lexington, MA; Amsterdam, the Netherlands; and Paris, France, June 22, 2021 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced it has entered into a definitive agreement to acquire Corlieve Therapeutics and its lead program, which will be known as AMT-260, to treat temporal lobe epilepsy, the most common form of focal epilepsy.

 

Corlieve’s lead gene therapy program employs miRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporal lobe epilepsy (TLE). TLE affects approximately 1.3 million people in the U.S. and Europe alone, of which approximately 800,000 patients are unable to adequately control acute seizures with currently approved anti-epileptic therapies. Patients with refractory TLE experience increased morbidity, excess mortality, and poor quality of life.

 

AMT-260 was originally developed by Corlieve in collaboration with Christophe Mulle, Ph.D., CNRS Research Director, at the Interdisciplinary Institute of Neurosciences, CNRS, University of Bordeaux, Valerie Crepel, Ph.D., Inserm Research Director at the Institut de Neurobiologie de la Méditerranée, INSERM, Aix-Marseille University, and REGENXBIO Inc. Drs. Mulle and Crepel will continue their collaborations with uniQure.

 

“The acquisition of Corlieve provides an extraordinary opportunity to transform the lives of hundreds of thousands of patients around the world suffering from epilepsy and aligns with our vision of pursuing unmet medical needs for disorders that impact large populations and can be addressed with gene therapies directed to the CNS and liver,” stated Matt Kapusta, Chief Executive Officer of uniQure. “The groundbreaking work of the Corlieve team, in collaboration with Drs. Mulle and Crepel and others, has led to compelling preclinical results in temporal lobe epilepsy that we believe can strategically leverage uniQure’s leading position in developing and delivering gene therapies that employ miRNA silencing technology. We look forward to welcoming the Corlieve team into the uniQure family as we join forces to advance this important and potentially transformative therapy into clinical studies.”

 

“I am very proud of what Corlieve has achieved in such a short amount of time. With the dedication and focus of our team and our collaboration partners, we have taken a promising therapeutic approach discovered by our scientific founders and created a potential transformative therapeutic opportunity for patients with refractory TLE,” said Richard Porter, Ph.D., founder and Chief Executive Officer of Corlieve. “As leaders in the field for miRNA gene therapy for neurological conditions, uniQure is the ideal long-term partner for us, and we look forward to working together to advance our program rapidly to the clinic for the benefit of the patients we serve.”

 

 

 

 

Upon the closing of the transaction, Dr. Porter will assume the role at uniQure of General Manager of the Corlieve subsidiary.

 

“As a founding investor, we are delighted to see that the translational work conducted by Corlieve is recognized by uniQure, further reinforcing our commitment to company creation based on partnering with the industry,” said Vanessa Malier, Managing Partner at Kurma Partners and Chairman of Corlieve.

 

Transaction Details

 

Under the terms of the agreement, uniQure will pay €46.3 million in an upfront payment of cash to acquire Corlieve.

 

Corlieve shareholders are eligible to receive the following additional payments of which up to 25% will be payable in uniQure ordinary shares at uniQure’s election: up to €43.7 million in development milestones through Phase I/II and €160 million in milestones associated with Phase III development and the approvals of AMT-260 in the U.S and European Union.

 

Corlieve has an established license and collaboration agreement with REGENXBIO that includes an exclusive license to AAV9 for the specific genetic target of AMT-260. Under the license and collaboration agreement, REGENXBIO received equity in Corlieve and is eligible to receive milestone payments and royalties on net sales of AMT-260.

 

The transaction has been approved by the Boards of both companies and does not require uniQure shareholder approval. The transaction is subject to customary closing conditions as well as review by the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) pursuant to articles L.151-3 and R.151-1 and seq. of the French Code Monétaire et Financier. Currently, the transaction is anticipated to be completed early in the third quarter of 2021.

 

SVB Leerink LLC is acting as sole financial advisor and Morgan Lewis is acting as legal advisor to uniQure. McDermott Will & Emery is acting as legal advisor to Corlieve.

 

About Corlieve Therapeutics 

 

Corlieve Therapeutics is a biotechnology company focused on bringing novel therapeutic options to patients with severe neurological disorders. The lead project is targeting aberrantly expressed kainate receptors in the hippocampus of patients with refractory TLE using a gene therapy approach. Corlieve was founded by Kurma Partners 4Q2019 on the basis of a partnership with REGENXBIO Inc., SATT Aquitaine Science Transfert, and Inserm Transfert. Corlieve is supported by its investors and partners Kurma Partners, Eurazeo, Pureos Bioventures, SATT Aquitaine Science Transfert, Inserm Transfert, Inserm CNRS, and REGENXBIO. For more information, please visit www.corlieve.com.

 

About uniQure 

 

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

 

 

 

 

uniQure Forward-Looking Statements

 

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether uniQure or Corlieve will advance the AMT-260 program to the clinic rapidly or at all, and whether the transaction is completed early in the third quarter of 2021 or ever. uniQure’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our risks associated with the integration of Corlieve and the execution of the AMT-260 development efforts, regulatory review of the transaction, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure’s Quarterly Report on Form 10-Q filed on May 10, 2021. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

 

Corlieve Contacts:

 

Amy Conrad 

Juniper Point

858-366-3243

amy@juniper-point.com 

Or

contact@corlieve.com

 

uniQure Contacts:    
     
FOR INVESTORS:   FOR MEDIA:
     
Maria E. Cantor Chiara Russo Tom Malone
Direct: 339-970-7536 Direct: 617-306-9137 Direct: 339-970-7558
Mobile: 617-680-9452 Mobile: 617-306-9137 Mobile:339-223-8541
 m.cantor@uniQure.com  c.russo@uniQure.com t.malone@uniQure.com

 

 

Exhibit 99.2

 

 

uniQure Announces Positive 52-Week Clinical Data from HOPE-B Pivotal Trial of
Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B 

and Provides Regulatory Update

 

~ Sustained increases in Factor IX (FIX) levels with mean FIX activity of 41.5 percent of normal in full
study population one year following a single administration of etranacogene dezaparvovec ~

 

~ Held pre-BLA submission meeting with FDA and aligned on primary endpoint analysis ~

 

Lexington, MA and Amsterdam, the Netherlands, June 22, 2021 — uniQure N. V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)- based gene therapy for the treatment of patients with severe and moderately severe hemophilia B. These are the first clinical data to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of hemophilia B patients receiving a single gene therapy investigational product to date.

 

Data from the HOPE-B pivotal study showed that participants continued to demonstrate durable, sustained increases in Factor IX (FIX) activity at 52-weeks post-infusion with a mean FIX activity of 41.5 percent of normal, as measured by a one-stage APTT-based clotting assay, compared to a mean FIX activity of 39.0 percent of normal at 26-weeks of follow-up. There continued to be no clinically significant correlation between pre-existing neutralizing antibodies to AAV5 (NAbs) and FIX activity in patients with NAb titers up to 678.2, a range expected to include more than 93 percent of the general population.

 

During the 52-week period, a single dose of etranacogene dezaparvovec significantly reduced the annualized rate of bleeding requiring treatment by 80 percent from a prospectively collected 3.39 at baseline to 0.68 bleeding episodes per year (p-value <0.0001). The annualized rate of spontaneous bleeding requiring treatment was also significantly reduced by 85 percent from a prospectively collected 1.16 at baseline to 0.18 bleeds per year during the 52-week period (p-value <0.0001).

 

Usage of FIX replacement therapy (IU/year and infusions/year) in all patients declined 96 percent during the 52-week period, with 52 of 54 patients (96 percent) successfully discontinuing their prophylactic infusions. As previously announced, of the two non-responders, one patient only received a partial dose (less than 10 percent of the dosage) due to an infusion reaction and a second patient had an unusually high pre-existing NAb titer of 3,212, which is expected in less than 1 percent of the general population.

 

Etranacogene dezaparvovec continues to be generally well-tolerated with no treatment-related serious adverse events. No inhibitors to FIX have been reported and no consistent relationship between safety and pre-existing NAb titers has been observed.

 

 

 

 

“We continue to be very encouraged by the data generated from the HOPE-B pivotal study of etranacogene dezaparvovec, which have been accepted for presentation at the annual International Society on Thrombosis and Haemostasis congress taking place next month” stated Ricardo Dolmetch, Ph.D., president of research and development at uniQure. “The 52-week data show mean FIX activity in the normal range and increase our confidence in the potential durability and long-term benefits of etranacogene dezaparvovec, bringing us one step closer to our goal of delivering this groundbreaking therapy to fulfill an unmet medical need for patients living with hemophilia B.”

 

Regulatory Update

 

The Company and its partner, CSL Behring, have had recent communications with U.S. Food and Drug Administration (FDA), including a pre-biologics licensing application (BLA) submission meeting held on June 4, 2021. The FDA confirmed that the primary evidence of durability of effect to inform regulatory decision-making will come from patients followed for at least a 52-week period beginning when etranacogene dezaparvovec-derived FIX levels have achieved steady state, rather than when etranacogene dezaparvovec is administered. This feedback was based upon review of statistical analysis plans, as no clinical data was provided or discussed. All patients in the HOPE-B pivotal study achieved steady-state FIX activity levels by 26-weeks after administration of etranacogene dezaparvovec. As a result, uniQure will now conduct as the sole primary endpoint a non-inferiority analysis of annualized bleeding rates (ABR) at 78 weeks after the administration (approximately 52-weeks after steady-state is achieved). The Company expects all patients to complete their 78 -week follow-up visits by the end of the third quarter of 2021, and the Company and CSL Behring expect to submit the BLA in first quarter of 2022.

 

About the HOPE-B Pivotal Clinical Trial

 

The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Fifty-four adult hemophilia B patients classified as severe or moderately severe (defined as less than or equal to 2% of normal FIX activity) and requiring prophylactic FIX replacement therapy were enrolled in a prospective, six-month observational period during which time they continued to use their current standard of care therapy to establish a baseline ABR. After the six-month lead-in period, patients received a single intravenous administration of etranacogene dezaparvovec at the 2x1013 gc/kg dose. Patients were not excluded from the trial based on their pre-existing NAbs to AAV5. Forty- three percent of patients in the study had pre-existing NAbs to AAV5 up to a maximum observed pre-dosing titer of over 3,200.

 

About Etranacogene Dezaparvovec

 

Etranacogene dezaparvovec consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicine (PRIME) regulatory initiative by the European Medicines Agency. uniQure and CSL Behring entered into a commercialization and license agreement providing CSL Behring exclusive global commercialization rights to etranacogene dezaparvovec. The collaboration combines uniQure’s differentiated gene therapy candidate in hemophilia B and CSL Behring’s strong global reach and commercial infrastructure in hematology in an effort to accelerate access of etranacogene dezaparvovec to hemophilia B patients around the world.

 

AAV5-based gene therapies have been demonstrated to be safe and well tolerated in a multitude of clinical trials, including being well-tolerated to date in six uniQure trials conducted in nearly 90 patients in hemophilia B and other indications. No patient treated in clinical trials with the uniQure’s AAV5 gene therapies has experienced any confirmed cytotoxic T-cell-mediated immune response to the capsid. Additionally, pre-clinical and clinical data show that AAV5-based gene therapies may be viable treatments in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates.

 

 

 

 

About uniQure

 

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

 

uniQure Forward-Looking Statements

 

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether patients in the Hope-B pivotal trial will complete their 78-week follow-up visits by the end of the third quarter of 2021, whether CSL Behring will submit a BLA for etranacogene dezaparvovec in the first quarter of 2022, whether the clinical data proves to be meaningful for the long-term outlook for hemophilia gene therapy or etranacogene dezaparvovec, whether etranacogene dezaparvovec has the potential to provide well-tolerated, long-term clinical benefits, and whether AAV5-based gene therapies can provide clinical benefit to patients with pre-existing neutralizing antibodies. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our Commercialization and License Agreement with CSL Behring, our and our collaborators’ clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure’s periodic securities filings, including is Annual Report on Form 10-K filed March 2, 2020 and Quarterly Report on Form 10-Q filed on May 10, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

 

uniQure Contacts

 

FOR INVESTORS:   FOR MEDIA:
     
Maria E. Cantor Chiara Russo Tom Malone
Direct: 339-970-7536 Direct: 617-306-9137 Direct: 339-970-7558
Mobile: 617-680-9452 Mobile: 617-306-9137 Mobile:339-223-8541
m.cantor@uniQure.com c.russo@uniQure.com t.malone@uniQure.com

 

 

 

 



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